Type 1 Gaucher Disease Clinical Trial
Official title:
An Open-Label Study to Assess the Pharmacokinetics, Safety, and Pharmacodynamics of Repeated Doses of Orally Administered AT2101 in Adult Patients With Type 1 Gaucher Disease
This is an open-label study designed to assess if AT2101 is safe in patients with Gaucher
disease and how AT2101 gets through the body after it is taken by mouth. The study is being
offered to adult patients with type 1 Gaucher disease who are currently receiving a stable
dose of enzyme replacement therapy (ERT) with imiglucerase. During the study, subjects will
not be receiving ERT (up to 35 days).
The study consists of a screening period (~14 days), a treatment period (12 days) and a
follow-up period (7 days after last dose). At two points in the study, subjects will be
housed in an in-patient treatment facility for 3 days/2 nights to accommodate all necessary
blood draws.
Status | Terminated |
Enrollment | 1 |
Est. completion date | July 2009 |
Est. primary completion date | July 2009 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | 18 Years to 65 Years |
Eligibility |
Inclusion Criteria: - Have a confirmed diagnosis of type 1 Gaucher disease with a documented gene mutation; - Clinically stable and on a stable dose of ERT for at least 2 years before study entry, with no changes to dose level or regimen in the last 6 months; - Willing to stop ERT for the duration of the study; - Male or female between the ages of 18 to 65 inclusive; - All subjects of reproductive potential are required to practice an acceptable method of contraception; - All subjects must have a body mass index of less than 30; and - Provide written informed consent to participate in the study. Exclusion Criteria: - A clinically significant disease, severe complications from Gaucher disease, or serious intercurrent illness that may preclude participation in the study in the opinion of the Investigator; - During the screening/wash out period, any clinically significant findings, based on physical exam, medical history review, lab assessment, vital sign assessment and/or other significant finding which would compromise the safety of the subject, or preclude the subject from completing the study as deemed by the Investigator; - Partial or total splenectomy; - History of pulmonary hypertension or Gaucher-related lung disease; - History of allergy or sensitivity to the study drug or any excipients, including any prior serious allergic reaction to iminosugars; - Screening or Day 1 (before dosing) 12-lead ECG demonstration QTc >450 msec in males or >470 in females; - Pregnant or breast-feeding; - Current/recent drug or alcohol abuse within the past 12 months; - Treatment with any investigational product, including investigational forms of ERT, in the 90 days before study entry; - Treatment in the previous 90 days with any drug known to have a well-defined potential for toxicity to a major organ; - Presence or sequelae of gastrointestinal, liver or kidney disease, or other conditions known to interfere with the absorption, distribution, metabolism, or excretion of drugs; or - Subject is otherwise unsuitable for the study in the opinion of the Investigator. |
Allocation: Non-Randomized, Endpoint Classification: Pharmacokinetics Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
United Kingdom | Guy's Drug Research Unit | London | |
United States | Comprehensive Phase One | Miramar | Florida |
Lead Sponsor | Collaborator |
---|---|
Amicus Therapeutics |
United States, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | To evaluate single-dose and multiple-dose pharmacokinetics of isofagomine following the oral administration of AT2101 in patients with type 1 Gaucher disease | Multiple times for up to three days following Day 1 and Day 10 | No | |
Secondary | To evaluate the safety and tolerability of oral AT2101 administered in patients with type 1 Gaucher disease | Daily | Yes | |
Secondary | To evaluate the pharmacodynamic effect of WBC GCase of oral AT2101 administered in patients with type 1 Gaucher disease. | Days 1, 5 & 7 (optional), 10 and 17 | No |
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